Wainua-Eplontersen
Wainua is a subcutaneous injection designed for adults managing nerve damage caused by hereditary transthyretin-mediated amyloidosis (ATTRv‑PN). It works via an antisense therapy that reduces the abnormal protein buildup damaging the nerves.
Voydeya-Danicopan
Voydeya is an oral complement factor D inhibitor added to standard therapy in adults with paroxysmal nocturnal hemoglobinuria. It helps reduce ongoing hemolysis outside blood vessels, improving anemia.
Fabhalta-Iptacopan
Fabhalta is an oral capsule that helps treat rare blood and kidney diseases by inhibiting complement factor B. It’s approved for adults with paroxysmal nocturnal hemoglobinuria (PNH), and to reduce proteinuria in complement 3 glomerulopathy (C3G) and IgA nephropathy.
Filsuvez-Birch Triterpenes
Filsuvez is a topical gel used on open wounds in patients with dystrophic or junctional epidermolysis bullosa (EB). It’s the first treatment that supports skin healing in this condition by promoting wound closure.
Zilbrysq-Zilucoplan
Zilbrysq is a once-daily self-administered injection for adults with generalized myasthenia gravis who test positive for anti-AChR antibodies. It blocks the complement C5 protein to reduce muscle weakness and improve strength.
Bimzelx-Bimekizumab
Bimzelx is a subcutaneous injection used to treat plaque psoriasis, psoriatic arthritis, axial spondyloarthritis (including ankylosing spondylitis), and hidradenitis suppurativa. It targets and blocks IL‑17A and IL‑17F cytokines to reduce inflammation and skin/joint symptoms.
Agamree-Vamorolone
Agamree is an oral suspension for Duchenne muscular dystrophy in children aged 2 and older. It’s a steroid-type medicine that helps protect muscles and slow disease progression.
Omvoh-Mirikizumab-Mrkz
Omvoh is a once-daily oral antibody for adults with moderately to severely active ulcerative colitis. It targets inflammatory pathways to reduce bowel inflammation and improve symptoms.
Loqtorzi-Toripalimab-Tpzi
Loqtorzi is an IV antibody used alongside other treatments for recurrent or metastatic nasopharyngeal carcinoma. It helps the immune system recognize and attack cancer cells.
Fruzaqla-Fruquintinib
Fruzaqla is a once-daily oral treatment for metastatic colorectal cancer in adults who have already tried other therapies. It inhibits VEGF receptors to slow tumor growth and improve survival.
